VectorSelector

ScientificInformation
ScientistsrefertoDNAdeliveryvehiclesasvectors.Eachvectorisdesignedtotargetspecific
cells.Traditionally,vectorshavebeenderivedfromviruses,includingretroviruses,
adenoviruses,adenoassociatedviruses,andherpessimplexviruses.Componentsofthevirus
thatcausediseaseareremovedandthegenetheresearcherwantstobedeliveredisinserted.
ThetransferofDNAintoacellbyamodifiedinfectiousvirusiscalledtransfection.This
distinguishesitfrominfection,bywhichavirusinsertsitsownDNAorRNAintoacell.Nonviral
genedeliveryapproachesbeingexaminedincludetheuseofliposomes,(lipidbasedpockets
thatcancarryplasmidDNA)orsimplynakedDNAwithnocarrier.Eachvectorandmethodof
deliveryvariesdependingonthespecificdisorderthetherapyaimstotreat.Threeimportant
factorstoconsiderwhenchoosingavectorforgenetherapyare:
1. Willthegenefitintothevector?
2. Willthevectortargettherighttypeoftissue?
3. Doesthevectorworkwithintheappropriatecellcyclestagetoaffectthedisease?

Therearetwobasicwaystodeliverageneintoagroupofcellsinapatientsbody.Thefirstis
toinjectthevectorintothebodyandspecificallytargetthosecells.Thisiscalledaninvivo
approach.

Thesecondway,calledexvivo,istodeliverthegenetocellswhiletheyare
outsidethebodyusingthefollowingprocedure:
1. Isolatethedesiredcellsfromthebody.
2. CulturethecellsinaPetridish,inthelaboratory.
3. Deliverthegenetothecells(usinganappropriatevector),activateit,andmakesureit
integratesproperlyintothecells.
4. Putthegeneticallymodifiedcellsbackintotheproperplaceinthebody,ensurethatthey
survivethere,andletthemgettowork.

Eachofthevectorsandmethodshasitsstrengthsandlimitationsforgenedelivery.Successin
genetherapydependsontheefficientdeliveryofthecorrectgenetothecorrectcellsinthe
correcttissue.Thus,theselectionofaparticularvectorishighlydependentuponitsproperties
andthecharacteristicsofthediseaseundergoingtreatment.

Assignment
Actingasemployeesofavectorsupplycompany,studentsresearchadiseasethatisa
candidateforgenetherapyanddeterminethebestvectortouse.Theymustthenpresenttheir
choiceandthereasoningbehinditinordertohelpsellthevectortoabiotechcompany
developingatherapyforthisdisease.

Assessment

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